GC Biopharma offers lysosomal storage disorder drug development updates at WORLD Symposium 2025
GC Biopharma said it presented updates on its lysosomal storage disorder (LSD) treatment development at the WORLD Symposium 2025, held in San Diego, U.S., from Feb. 3 to 7.
WORLD Symposium is an international forum where experts in lysosomal storage diseases share the latest insights and research to develop better treatment options. At this year’s event, GC Biopharma presented preclinical findings on its investigational oral chaperone therapy GC2126A for GM1 gangliosidosis and its investigational treatment GC1130A for Sanfilippo syndrome type A (MPSIIIA) through poster sessions while also gathering insights on the latest research trends in rare diseases.
GM1 gangliosidosis is a rare, inherited neurodegenerative disorder caused by a genetic defect leading to a deficiency in beta-galactosidase enzyme activity. It occurs in about one in 100,000 newborns, with onset typically occurring before age six. The condition is characterized by neurodegeneration, seizures, and muscle weakness, and currently, no approved treatments are available.
During the poster session, GC Biopharma presented data demonstrating that its candidate drug GC2126A increased beta-galactosidase activity in a dose-dependent manner across various tissues, including the brain, in an animal model.
The preclinical study showed that a seven-day oral administration of GC2126A reduced the accumulation of GM1-ganglioside in the brain by more than 70 percent.
The company also presented preclinical findings on GC1130A, its investigational therapy for Sanfilippo syndrome type A (MPSIIIA). MPSIIIA is a severe autosomal recessive disorder caused by a genetic defect that causes the accumulation of heparan sulfate.
The disease primarily leads to progressive and severe neurological impairment, with most patients not surviving beyond their teenage years. Given the lack of approved treatments, a significant unmet medical need remains.
GC Biopharma is co-developing GC1130A as a first-in-class biologic in collaboration with Novel Pharma, a local biotech firm.
The therapy utilizes the company’s high-concentration protein formulation technology and is administered via intracerebroventricular (ICV) injection to enhance drug delivery to the brain.
The ICV delivery method was previously applied to GC Biopharma’s Hunter syndrome treatment, Hunterase, which received marketing approval in Japan. To advance the development of GC1130A, GC Biopharma, and Novel Pharma have secured investigational new drug (IND) approvals for phase 1 clinical trials in the U.S., Korea, and Japan and are conducting multinational clinical studies.
At the symposium, the company presented data indicating that ICV administration provided superior drug delivery efficiency compared to intrathecal (IT) injection, further supporting the potential efficacy of GC1130A.
“With our expertise and accumulated knowledge in lysosomal storage disorder treatment development, we will continue to expand our research into other rare diseases to provide new treatment options for patients in need,” GC Biopharma Medical Division Head Shin Soo-kyung.